In the final draft guidance rolled out by the National Institute for Health and Care Excellence- NICE, a single-dose therapy, eladocagene exuparvovec, has been recommended for patients who are more than 18 months old and happen to be suffering from serious aromatic L-amino acid decarboxylase- AADC deficiency.
Designed to rectify the underlying genetic defect when it comes to the condition, this happens to be the first gene therapy to be infused directly in the brain with a minimally invasive process. The clinical evidence goes on to show that it enhances the motor development and apparently these improvements can indeed be lasting long.
AADC which is generally caused due to a genetic mutation mainly affects the central nervous system which happens to be the body’s control system which regulates bodily functions like the heart rate, gastrointestinal issues as well as the endocrine system. When it comes to young children, normal motor development like head control, sitting, and walking is particularly affected.
Around 80% of the people suffering from AADC deficiency, have a serious form of this ultra-rare condition. It is well to note that, until now, there have been no treatments available. It is estimated that only 10 children in the UK happen to have this kind of disorder, with only a very small number being eligible for a treatment such as this.
According to Helen Knight who happens to be Director of Health Technology Assessment at NICE, the decision in the NICE final draft comes after a confidential commercial deal which has taken place between NHS England and one of the gene therapy companies.
It goes on to recognize the uncertainty in the evidence when it comes to eladocagene exuparvove’s long-term efficacy and has further enabled the committee to push it as a good value for NHS as well as taxpayers.
Knight further added that the committee of the organization agreed that the clinical trials when it came to gene therapy showed the potential for significant benefits that were sometimes absolutely life-changing.
It is expected that NICE will be publishing its final guidance when it comes to eladocagene exuparvove next month in April.
Apparently, the gene therapy went on to be approved in November last year by MHRA in Great Britain. The treatment has also been validated across 27 EU member states, as well as Iceland, Liechtenstein, Northern Ireland, and Norway.